Fingolimod was first approved for adult patients with the relapsing form of MS, and was granted Priority Review and Breakthrough Therapy designation in December.
The US Food and Drug Administration (FDA) has approved fingolimod (Gilenya) for an expanded indication that makes it the first treatment of multiple sclerosis (MS) in pediatric patients.
The therapy, from Novartis, was previously approved for the treatment of adult patients with relapsing MS. Its new indication approves it for the treatment of children and adolescents aged 10 years and older with the relapsing form of the disease. The FDA had previously granted Priority Review and Breakthrough Therapy designation for this indication.
Billy Dunn, MD, director of the Division of Neurology Products at the FDA Center for Drug Evaluation and Research, remarked on the significance of the historic approval.
“Multiple sclerosis can have a profound impact on a child’s life,” Dunn said in a statement. “This approval represents an important and needed advance in the care of pediatric patients with multiple sclerosis.”
According to the FDA, current estimates report that 8000-10,000 children and adolescents in the US suffer from MS, though most patients experience their first symptoms of the neurological condition between the ages of 20-40.
In a clinical trial testing the efficacy of fingolimod in treating pediatric patients with MS, researchers compared the drug to interferon beta-1a in 214 patients aged 10-17. After 24 months of treatment, 86% of patients receiving fingolimod remained free of relapses, while just 46% of patients treated with comparative therapy remained relapse-free.
Adverse effects of the therapy reported in the clinical trial — which included headache, liver enzyme elevation, diarrhea, cough, flu, and more — were similar to those reported by adult patients in previous studies.
In an interview with MD Magazine at the 7th joint meeting of the European and Americas Committees for Treatment and Research in Multiple Sclerosis (ECTRIMS) in Paris, France, last year, Tinuja Chitnis, MD, discussed the potential of fingolimod in pediatric patients — as indicated by the eventual application-accompanying results from the PARADIGM trial.
“Children [with MS] have a highly inflammatory disease with 2 to 3 times as many relapses as an adult patient will,” Chitnis said. “This study tells us that clearly there is probably an age-related effect to fingolimod and that it performs very well in young patients, performs very well in young adults, and in fact, these results were better than the overall adult population.”
Though fingolimod's safety profile was found to be similar — or even better — to that seen in adults with MS, Chitnis noted an imbalance in seizure rates of children treated with the oral drug during clinical trials.
“That’s an important factor to follow up on," Chitnis said.
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